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Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

2014-07-23 21:09:04 | BioPortfolio

Summary

This study will evaluate safety and clinical outcomes of treatment with Replagal in adult patients with Fabry disease who have completed Study TKT028.

Description

The objective of the study is to evaluate the long term safety and effect of continued dosing with Replagal (0.2 mg/kg administered intravenously [IV] every other week)following 53 weeks of treatment in Study TKT028 on the reduction from baseline in left ventricular mass (LVM) as measured by echocardiography

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Fabry Disease

Intervention

Replagal

Location

1st School of Medicine Charles University
Prague
Czech Republic

Status

Enrolling by invitation

Source

Shire Human Genetic Therapies, Inc.

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-07-23T21:09:04-0400

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PubMed Articles [14403 Associated PubMed Articles listed on BioPortfolio]

Phenotypic characteristics of the p.Asn215Ser (p.N215S) GLA mutation in male and female patients with Fabry disease: A multicenter Fabry Registry study.

The p.Asn215Ser or p.N215S GLA variant has been associated with late-onset cardiac variant of Fabry disease.

Hemizygous Fabry disease associated with membranous nephropathy: A rare case report
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Screening Test of Fabry Disease in Patients with Renal Replacement Therapy in the City of Modena.

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Genital angiokeratoma in a woman with Fabry disease: the dermatologist's role.

Fabry disease is a rare lysosomal storage disorder, inherited in an X-linked manner. It is characterized by the deficiency of the enzyme alpha-galactosidase, leading to a buildup of glycosphingolipids...

α-Galactosidase A-deficient rats accumulate glycosphingolipids and develop cardiorenal phenotypes of Fabry disease.

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Medical and Biotech [MESH] Definitions

An X-linked inherited metabolic disease caused by a deficiency of lysosomal ALPHA-GALACTOSIDASE A. It is characterized by intralysosomal accumulation of globotriaosylceramide and other GLYCOSPHINGOLIPIDS in blood vessels throughout the body leading to multi-system complications including renal, cardiac, cerebrovascular, and skin disorders.

Members of the class of neutral glycosphingolipids. They are the basic units of SPHINGOLIPIDS. They are sphingoids attached via their amino groups to a long chain fatty acyl group. They abnormally accumulate in FABRY DISEASE.

Glycosphingolipids which contain as their polar head group a trisaccharide (galactose-galactose-glucose) moiety bound in glycosidic linkage to the hydroxyl group of ceramide. Their accumulation in tissue, due to a defect in ceramide trihexosidase, is the cause of angiokeratoma corporis diffusum (FABRY DISEASE).

Animate or inanimate sources which normally harbor disease-causing organisms and thus serve as potential sources of disease outbreaks. Reservoirs are distinguished from vectors (DISEASE VECTORS) and carriers, which are agents of disease transmission rather than continuing sources of potential disease outbreaks.

Analyses for a specific enzyme activity, or of the level of a specific enzyme that is used to assess health and disease risk, for early detection of disease or disease prediction, diagnosis, and change in disease status.

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